STOCK TITAN

Rocket Pharmaceuticals enters up to $150M loan deal

Rocket said existing resources and the initial draw are expected to fund planned operations into the third quarter of 2028.

(High)

Sentiment and the balance of points

Rhea-AI Sentiment reads the wording of the document, how positive or negative its language is on a 1 to 5 scale. The balance of points shown with the takes weighs what the document actually discloses, so the two can disagree, for example when a trial that missed its main goal is described in upbeat language.

Form Type
8-K

Rhea-AI Filing Summary

Rocket Pharmaceuticals entered a senior secured term-loan facility with Hercules Capital and other lenders for up to $150.0 million; $35.0 million was funded on September 30, 2026. Further advances include borrower-option tranches, $30.0 million after a clinical milestone, and a final tranche of up to $50.0 million subject to lender investment committee approval.

Interest is the greater of prime or 6.75%, plus 2.40% annually; payments are interest-only through April 1, 2029, with milestone-based extensions. Maturity is October 1, 2030, extendable to October 1, 2031 if the milestone is achieved and no default continues. Substantially all borrower assets secure the loans; a qualified-cash covenant requires 35%–75% of outstanding loans and is not tested when market capitalization exceeds $600.0 million.

Rocket also issued warrants to purchase up to 1,755,853 shares at $2.99 each; 409,699 shares were exercisable after the initial advance, with full exercisability tied to a maximum facility draw. The RP-A501 update reported three Cohort 4 patients treated at the recalibrated dose, with one resolved Grade 3 GGT increase. Cohort 3 was discontinued after serious adverse events in two patients on a C3-inhibitor regimen; pivotal topline results are provisionally targeted for mid-2028.

1 point · 0 major

How this balance works

Rhea-AI gives every point it takes from this document a weight. Minor counts 1, Moderate 3 and Major 9, so one Major point outweighs several Minor ones. The bar adds up the weights on each side, and when neither side holds more than 65% of the total the balance reads Mixed.

It reads the document as published, with the same rules for every company, and it does not look at what the market expected or at how the stock traded, so a point can be objectively good on a day the stock falls.

Rhea-AI Sentiment measures something else, the tone of the wording.

0 major · 1 point

Hollow bars mark forward-looking points. How the balance works

Positive

  • Moderate point. Forward-looking: it has not happened yet and may not happen.Rocket expects resources and the initial draw to fund operations into Q3 2028.

Negative

  • Moderate pointCohort 3 was discontinued after serious adverse events in 2 patients.

Filing Explained

Rocket’s stated runway reaches the third quarter of 2028; further borrowing is conditional, and warrant exercise could dilute existing holders.

With the facility’s initial $35 million funded, Rocket says its June 30, 2026 cash, cash equivalents and investments of $283.7 million, together with that advance, are expected to support planned operations into Q3 2028; further draws could extend the estimate into 2029, subject to conditions, timing and spending.

Warrants for 409,699 shares are currently exercisable; exercising them would add shares and reduce existing holders’ percentage ownership, absent offsetting changes.

Item 1.01 Entry into a Material Definitive Agreement Business
The company signed a significant contract such as a merger agreement, credit facility, or major partnership.
Item 2.03 Creation of a Direct Financial Obligation or an Obligation under an Off-Balance Sheet Arrangement Financial
The company incurred a new significant debt or off-balance-sheet obligation.
Item 3.02 Unregistered Sales of Equity Securities Securities
The company sold equity securities in a private placement or other unregistered transaction.
Item 7.01 Regulation FD Disclosure Disclosure
Material non-public information disclosed under Regulation Fair Disclosure, often investor presentations or guidance.
Item 9.01 Financial Statements and Exhibits Exhibits
Financial statements, pro forma financial information, or exhibit attachments filed with this report.
Term-loan facility Up to $150.0 million Aggregate principal amount available across five tranches
Initial funded advance $35.0 million Tranche 1-A funded on September 30, 2026
Annual interest rate Greater of prime rate or 6.75%, plus 2.40% Floating rate on the term loans
Initial maturity October 1, 2030 May extend to October 1, 2031 upon milestone achievement and while no default continues
Warrant shares Up to 1,755,853 common shares Warrants issued to the lenders; full exercisability depends on drawing the maximum facility amount
Warrant exercise price $2.99 per share Exercise price for the warrants
Expected operating runway Into the third quarter of 2028 Company expectation based on current operating plan, existing resources, and the initial $35 million advance
minimum cash covenant financial
"minimum cash covenant"
A minimum cash covenant is a loan agreement clause that requires a company to keep at least a specified amount of cash or liquid assets on hand, like a bank requiring you to maintain a minimum balance. It matters to investors because it limits how management can spend or return cash, reduces the risk of surprise default by ensuring a short-term safety buffer, and can signal lender concern about the company’s liquidity.
end of term charges financial
"end of term charges payable upon the earliest to occur of the Maturity Date"
Biologics License Application regulatory
"FDA approval of a Biologics License Application for RP-A501"
A biologics license application is a formal request submitted to regulatory authorities seeking approval to market a new biological medicine, such as vaccines or treatments made from living organisms. It is a comprehensive review process that evaluates the safety, effectiveness, and manufacturing quality of the product. For investors, receiving approval signals that a biological therapy can be sold to the public, potentially leading to revenue growth and market success.
immunomodulatory regimen medical
"revised immunomodulatory regimen"
net issuance (cashless) basis financial
"on a net issuance (cashless) basis"

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

How much did RCKT receive under the Hercules loan facility?

Rocket received $35.0 million at closing under a facility of up to $150.0 million. The agreement includes a $20.0 million Tranche 1-B advance available at the borrower’s option through June 30, 2027, and a $15.0 million Tranche 1-C advance available after Tranche 1-B is fully drawn or expires, through September 30, 2027. A $30.0 million tranche depends on achievement of the Tranche 2 milestone.

What are the exercise terms for RCKT’s Hercules warrants?

Rocket issued warrants to purchase up to 1,755,853 common shares at $2.99 per share. After the initial advance, warrants for 409,699 shares were exercisable. The warrants may be exercised for cash or, at each holder’s election, on a net issuance (cashless) basis; they expire at the earliest of the seventh anniversary of the closing date, certain cash acquisitions, or full exercise.

What is RCKT’s RP-A501 Phase 2 efficacy target?

The stated target is at least 7 of 12 responders at 12 months. Each responder must meet both criteria: myocardial LAMP2 protein expression improvement of at least Grade 1 from baseline and a reduction in left ventricular mass index of at least 10% from baseline.

How long does RCKT expect its cash runway to last?

Based on its current operating plan, Rocket expects its cash, cash equivalents and investments of $283.7 million as of June 30, 2026, together with the initial $35 million facility advance, to fund planned operations into the third quarter of 2028. Additional facility borrowings could extend the runway into 2029, subject to borrowing conditions, draw timing and amounts, and operating expenditures.

What safety findings did RCKT report for RP-A501 Cohort 4?

The update reported three patients treated at the recalibrated dose. One patient had a Grade 3 increase in gamma-glutamyltransferase that resolved; all three patients were discharged within one to two weeks after infusion. The presentation reported no evidence of thrombotic microangiopathy, capillary leak, or other complement-mediated events through three months.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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Learn about SEC filing dates

UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, DC 20549

FORM 8-K

CURRENT REPORT
Pursuant to Section 13 OR 15(d) of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): September 30, 2026



Rocket Pharmaceuticals, Inc.
(Exact name of registrant as specified in its charter)



Delaware
001-36829
04-3475813
(State or other jurisdiction of incorporation)
(Commission File Number)
(IRS Employer Identification No.)



9 Cedarbrook Drive, Cranbury, NJ
 
08512
(Address of principal executive offices)
 
(Zip Code)



Registrant’s telephone number, including area code: (646) 440-9100



Not applicable
(Former name or former address, if changed since last report)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2):

☐
Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐
Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐
Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐
Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading
Symbol(s)

Name of each exchange on which
registered
Common stock, $0.01 par value

RCKT

The Nasdaq Global Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐



Item 1.01.
Entry into a Material Definitive Agreement.

Loan and Security Agreement

On September 30, 2026, Rocket Pharmaceuticals, Inc. (the “Company”), together with its subsidiaries Spacecraft Seven, LLC and Zebrafish Merger Sub II, LLC (together with the Company, “Borrower”), entered into a Loan and Security Agreement (the “Loan Agreement”) with the several banks and other financial institutions or entities from time to time party thereto (collectively, the “Lenders”) and Hercules Capital, Inc., a Maryland corporation (“Hercules”), in its capacity as administrative agent and collateral agent for itself and the Lenders (in such capacities, the “Agent”), providing for up to five tranches of senior secured term loans in an aggregate principal amount of up to $150.0 million (the “Term Loans”).

The Term Loans are available in the following tranches: (i) a Tranche 1-A advance of $35.0 million, which was funded in full on September 30, 2026 (the “Closing Date”); (ii) a Tranche 1-B advance of $20.0 million, available at Borrower’s option at any time through June 30, 2027; (iii) a Tranche 1-C advance of $15.0 million, available at Borrower’s option during the period beginning upon the earlier of the full draw or expiration of the Tranche 1-B commitment and ending September 30, 2027; (iv) a Tranche 2 advance of $30.0 million, available at Borrower’s option following Borrower’s achievement of the Tranche 2 Milestone (as defined in the Loan Agreement) through the earlier of December 15, 2028 and sixty (60) days following achievement of such milestone; and (v) a Tranche 3 advance of up to $50.0 million, available at Borrower’s option, subject to the Lenders’ investment committee approval in its sole discretion, during the period following the earlier of the full draw or expiration of the Tranche 2 commitment (or, if earlier, December 15, 2028) through the amortization date described below.

The Term Loans mature on October 1, 2030, subject to extension to October 1, 2031 upon Borrower’s achievement of the Tranche 2 Milestone, so long as no default or event of default has occurred and is continuing (as so extended, the “Maturity Date”).

The Term Loans bear interest at a floating per annum rate equal to the sum of (x) the greater of (i) prime rate and (ii) 6.75%, plus (y) 2.40%. Borrowings under the Loan Agreement are interest-only through April 1, 2029, which period may be extended to April 1, 2030 if Borrower achieves the Tranche 2 Milestone by such date, and may be further extended to October 1, 2030 if Borrower additionally achieves an approval milestone relating to FDA approval of a Biologics License Application for RP-A501 for the treatment of Danon Disease (the “Approval Milestone”), in each case so long as no default or event of default has occurred and is continuing. Following the applicable interest-only period, borrowings are repayable in equal monthly installments of principal and interest through the Maturity Date. Upon the occurrence and during the continuation of an event of default, outstanding obligations bear interest at the otherwise applicable rate plus 4.00% per annum.

Borrower may voluntarily prepay the Term Loans in whole or in part, subject to a prepayment charge equal to (i) 3.00% of the principal amount prepaid if prepaid prior to the first anniversary of the Closing Date, (ii) 2.00% if prepaid on or after the first anniversary but prior to the second anniversary of the Closing Date, and (iii) 1.00% if prepaid on or after the second anniversary of the Closing Date and prior to the Maturity Date.

In connection with the closing of the Tranche 1-A advance, Borrower paid a customary initial facility fee and due diligence fee. Borrower will also pay a customary facility fee upon each advance under Tranche 1-B, Tranche 1-C, Tranche 2 and Tranche 3. The Loan Agreement also provides for end of term charges payable upon the earliest to occur of the Maturity Date, repayment in full, partial prepayment or acceleration of the obligations, in an amount equal to a percentage of the principal amount of each advance being repaid or prepaid, or that otherwise becomes due and payable, which percentage varies depending on when such repayment, prepayment or acceleration occurs relative to the Closing Date.

Borrower’s obligations under the Loan Agreement are secured by a first-priority security interest in substantially all of the assets of Borrower, including its intellectual property, subject to customary exceptions. The Loan Agreement contains a minimum cash covenant, tested beginning on a specified test date (which may be deferred based on the amount of net cash proceeds of qualified equity issuance  received by the Company after the Closing Date), requiring Borrower to maintain qualified cash equal to a specified percentage of the outstanding Term Loans (ranging from 35% to 75% depending on whether the Tranche 2 Milestone and the Approval Milestone have been achieved), which covenant is not tested at any time the Company’s market capitalization exceeds $600.0 million.


The Loan Agreement also contains customary representations and warranties and affirmative and negative covenants, including restrictions on indebtedness, liens, investments, mergers, dispositions, distributions and transactions with affiliates, subject to certain exceptions, as well as customary events of default, including payment defaults, breach of covenants, breach of representations and warranties, cross-defaults, bankruptcy-related defaults, judgment defaults and the occurrence of a material adverse effect. Upon the occurrence of an event of default, the Agent and the Lenders may declare all obligations under the Loan Agreement immediately due and payable and exercise other remedies available to them as secured creditors of Borrower.

Warrant Agreements

On September 30, 2026, in connection with the Loan Agreement, and in consideration for the financial accommodations made by the Lenders and the Agent in the Loan Agreement, the Company issued warrants to the Lenders to purchase up to an aggregate of 1,755,853 shares of the Company’s common stock, par value $0.01 per share (“Common Stock”), at an exercise price of $2.99 per share (the “Warrants” and each, a “Warrant”).

Each Lender’s Warrant becomes exercisable, and remains exercisable, for a number of shares of Common Stock determined by multiplying the maximum number of shares subject to such Warrant by a fraction, the numerator of which is the aggregate original principal amount of Term Loan advances funded under the Loan Agreement by the applicable Lender and the denominator of which is the total principal amount of Term Loans committed to by that Lender, such that the Warrants become exercisable in full only if the Company draws the maximum $150.0 million available under the Loan Agreement. Following the closing of the Tranche 1-A advance, warrants to purchase an aggregate of 409,699 shares are exercisable.

The Warrants are exercisable, in whole or in part, at any time prior to the earliest to occur of (i) the seventh anniversary of the date of Closing Date, (ii) the consummation of certain cash acquisitions of the Company, and (iii) exercise of the applicable Warrant in full. The exercise price and the number of shares issuable under the Warrants are subject to customary adjustment for stock splits, combinations, reclassifications, dividends and similar events, as well as adjustment upon certain acquisition transactions. The Warrants may be exercised for cash or, at each holder’s election, on a net issuance (cashless) basis.

The foregoing descriptions of the Loan Agreement and the Warrants do not purport to be complete and are qualified in their entirety by reference to the full text of the Loan Agreement and the form of Warrant Agreement, copies of which are filed as Exhibits 10.1 and 10.2, respectively, to this Current Report on Form 8-K and are incorporated herein by reference.

Item 2.03.
Creation of a Direct Financial Obligation or an Obligation Under an Off-Balance Sheet Arrangement of a Registrant.

The information set forth under Item 1.01 above is incorporated by reference into this Item 2.03.

Item 3.02.
Unregistered Sales of Equity Securities.

The information set forth under Item 1.01 above regarding the issuance of the Warrants is incorporated by reference into this Item 3.02. The Warrants and the shares of Common Stock issuable upon exercise of the Warrants have not been and will not be registered under the Securities Act of 1933, as amended (the “Securities Act”), and were issued in reliance on the exemption from registration provided by Section 4(a)(2) of the Securities Act, as a transaction by an issuer not involving a public offering. The Lenders represented that they were “accredited investors” as defined in Regulation D under the Securities Act and that they were acquiring the Warrants for investment purposes and not with a view toward distribution.

Item 7.01.
Regulation FD Disclosure.

On October 6, 2026, the Company issued a press release announcing the entry into the Loan Agreement with Hercules, a copy of the which is furnished as Exhibit 99.1 hereto.

Additionally, the Company prepared an investor presentation providing certain updates on the Company’s Danon Disease Program used in a corporate webinar on October 6, 2026, which is furnished as Exhibit 99.2 hereto and is incorporated herein by reference.


The information under this Item 7.01, including Exhibit 99.1 and Exhibit 99.2, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended, (the “Exchange Act”) or otherwise subject to the liabilities of that section, and shall not be deemed to be incorporated by reference into the filings of the Company under the Securities Act or the Exchange Act, except as shall be expressly set forth by specific reference in such filing.

Item 9.01.
Financial Statements and Exhibits.

(d)
Exhibits.

10.1*
Loan and Security Agreement, dated as of September 30, 2026, by and among Rocket Pharmaceuticals, Inc., Spacecraft Seven, LLC, Zebrafish Merger Sub II, LLC, the Lenders party thereto and Hercules Capital, Inc.
10.2
Form of Warrant Agreement
99.1
Press Release of Rocket Pharmaceuticals, Inc. dated October 6, 2026
99.2

Investor Presentation of Rocket Pharmaceuticals, Inc.

104
Cover Page Interactive Data File (embedded within the Inline XBRL document).

*
Certain portions of this exhibit (indicated by “[***]”) have been omitted pursuant to Item (601)(b)(10) of Regulation S-K.


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.


Rocket Pharmaceuticals, Inc.



Date: October 6, 2026
By:
/s/ Martin Wilson

 
Martin Wilson

 
General Counsel and Chief Corporate Officer




Exhibit 99.1


Rocket Pharmaceuticals Secures Strategic Credit Facility for Up to $150 Million from Hercules Capital
 
CRANBURY, N.J. – October 6, 2026 – Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated, commercial-stage biotechnology company advancing genetic medicines for rare and life-threatening diseases, focused on inherited cardiovascular disorders, today announced that it has entered into a credit facility agreement with Hercules Capital, Inc. (NYSE: HTGC) for up to $150 million. The facility strengthens Rocket’s financial position and provides access to additional capital to support execution of its pivotal Phase 2 study of RP-A501 for Danon disease and continued advancement of its broader cardiovascular pipeline.
 
“Building on the sale of our priority review voucher, this financing diversifies our sources of capital and strengthens our ability to execute and deliver on our cardiovascular strategy,” said Gaurav Shah, M.D., Chief Executive Officer of Rocket Pharmaceuticals. “The staged structure provides flexibility to align additional funding with progress across our programs. We remain focused on allocating capital thoughtfully, executing the pivotal Danon study and advancing genetic medicines for patients with serious inherited heart diseases”

“Rocket is advancing toward important milestones across its cardiovascular pipeline, led by the pivotal Danon program,” said R. Bryan Jadot, Senior Managing Director and Group Head of Life Sciences at Hercules Capital. “We are pleased to provide a flexible financing solution that supports the Company’s development priorities. We look forward to working alongside Rocket as it builds on its expertise in cardiovascular genetic medicines.”
 
Under the agreement, Rocket received $35 million at closing and may draw an additional $35 million at its option during specified periods, subject to the terms and conditions of the agreement. An additional $30 million may become available upon achievement of a specified Danon clinical milestone, and an additional $50 million is subject to Hercules’ investment committee approval. The facility has an initial 30-month interest-only period and a 48-month maturity, with extensions available upon achievement of specified milestones. The financing also includes warrants to purchase shares of Rocket’s common stock.
 
As of June 30, 2026, Rocket had cash, cash equivalents and investments of $283.7 million. Based on its current operating plan, Rocket expects these resources, together with the initial $35 million received under the facility, to fund planned operations into the third quarter of 2028. Additional borrowings under the facility could extend Rocket’s cash runway into 2029, subject to satisfaction of applicable borrowing conditions and depending on the timing and amount of future draws and the Company’s operating expenditures.
 
Additional information regarding the financing agreement will be disclosed in Rocket’s filings with the Securities and Exchange Commission.
 
LifeSci Capital served as financial advisor to Rocket on the term loan financing. PJT served as a capital markets advisor to Rocket.
 

About Rocket Pharmaceuticals, Inc.
 
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully integrated commercial-stage biotechnology company developing genetic medicines for rare and life-threatening diseases, with a strategic focus on inherited cardiovascular disorders and additional programs in hematology and immunology. Rocket's cardiovascular portfolio includes three clinical-stage gene therapy programs targeting hypertrophic, arrhythmogenic, and dilated cardiomyopathies, together representing one of the broadest pipelines focused on inherited heart disease. The Company’s integrated platform combines proprietary adeno-associated virus (AAV) manufacturing capabilities and extensive clinical experience in cardiac gene therapy.
 
For more information about Rocket, please visit www.rocketpharma.com and follow us on LinkedIn, YouTube, and X.

Rocket Cautionary Statement Regarding Forward-Looking Statements
This press release contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involve risks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause results to differ materially from those expressed or implied by such forward-looking statements. Rocket makes such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. All statements other than statements of historical fact contained in this release are forward-looking statements.

These forward-looking statements include, but are not limited to, statements concerning Rocket’s expected cash runway into the third quarter of 2028 and the potential to extend its cash runway into 2029 through additional borrowings under the credit facility; its ability to access and draw additional amounts under the facility, satisfy the conditions for future advances or extensions, and achieve applicable clinical and regulatory milestones; the anticipated use of proceeds; the expected benefits of the facility, including increased financial strength and strategic and operational flexibility; and Rocket’s plans to execute its pivotal Phase 2 study of RP-A501 for Danon disease and advance its broader cardiovascular pipeline.

Although Rocket believes that the expectations reflected in these forward-looking statements are reasonable, Rocket cannot guarantee such outcomes. Actual results may differ materially as a result of various important factors, including Rocket’s ability to satisfy the conditions for additional borrowings or extensions under the facility; the availability and timing of future advances, including Hercules’ investment committee approval of the final tranche; Rocket’s ability to comply with the facility’s covenants, including applicable minimum-cash requirements, and meet its debt service and repayment obligations; the results, timing and costs of Rocket’s ongoing and planned clinical trials; unexpected safety events; the timing and outcome of regulatory interactions and submissions; manufacturing and product-supply considerations; Rocket’s future capital requirements and ability to obtain additional funding; changes in its operating plan, development priorities, expenses or cash requirements; and other restrictions and obligations imposed by the credit facility. Additional risks are described under “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2025, filed with the Securities and Exchange Commission on February 26, 2026, and in its subsequent SEC filings, including its Quarterly Reports on Form 10-Q.

Accordingly, readers should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.


Investors & Media
Meg Dodge
mdodge@rocketpharma.com

Brooke Schuster
bschuster@rocketpharma.com




Exhibit 99.2

 RP-A501 Danon Disease Program Update  © 2026 Rocket Pharmaceuticals  Rocket Pharmaceuticals  October 6, 2026 
 

 DISCLAIMER  2  © 2026 Rocket Pharmaceuticals  This presentation contains forward-looking statements concerning Rocket’s future expectations, plans and prospects that involverisks and uncertainties, as well as assumptions that, if they do not materialize or prove incorrect, could cause actual results to differ materially from those expressed or implied by such forward-looking statements. Rocket makes these statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements often include words such as “believe,” “expect,” “anticipate,” “intend,” “plan,” “estimate,” “seek,” “will,” “may,” “suggest” or similar terms, variations of such terms or the negative of those terms.  These forward-looking statements include, but are not limited to, statements concerning Rocket’s cash runway and financial position; its ability to obtain additional funding to conduct planned research and development efforts; the potential safety and effectiveness of RP-A501, including at the recalibrated dose under the modified protocol; its ability to continue enrollment and dosing, complete the pivotal study and achieve its prespecified efficacy success criterion; the expected timing and results of ongoing and planned clinical trials; the expected timing and outcome of regulatory interactions and submissions, including a potential biologics license application seeking accelerated approval; Danon disease prevalence and addressable patient estimates; patient identification and referral efforts; potential commercial opportunity and revenue; commercialization plans, including the development of sales and marketing capabilities and relationships with treatment centers and other third parties; and potential development in additional patient populations.  Although Rocket believes that the expectations reflected in these forward-looking statements are reasonable, it cannot guarantee such outcomes. Actual results may differ materially as a result of various important factors, including, without limitation, clinical trial results and safety findings, including the risk of additional serious adverse events; Rocket’s ability to identify and enroll eligible patients and successfully complete clinical studies; the possibility that earlier clinical observations or nonclinical findings may not predict subsequent clinical outcomes; changes in regulatory requirements or expectations; Rocket’s ability to obtain regulatory approval and meet applicable post-approval requirements; manufacturing, product-quality and supply risks; dependence on third parties for development, manufacture, marketing, sales and distribution; the availability of sufficient funding and unexpected expenditures; the accuracy of population estimates and assumptions underlying commercial models; pricing,  reimbursement, market acceptance and competition; the outcome of litigation; Rocket’s ability to achieve the expected benefits of its portfolio prioritization and strategic restructuring; and its ability to obtain and enforce patents and defend against third-party infringement claims. Additional risks are described in the section entitled “Risk Factors” in Rocket’s Annual Report on Form 10-K for the year ended December 31, 2025, filed February 26, 2026 with the Securities and Exchange Commission, and subsequent filings with the SEC, including its Quarterly Reports on Form 10-Q.  RP-A501 is investigational, and its safety and efficacy have not been established. Clinical observations presented here are based on small, uncontrolled studies with varying follow-up. Preliminary findings may change as additional data become available, and results from earlier cohorts may not predict outcomes at the recalibrated dose under the modified protocol. Meeting the pivotal study’s efficacy success criterion would not, by itself, establish that RP-A501 will receive regulatory approval. FDA’s assessment would consider the complete application, including safety, efficacy, manufacturing and product-quality information. Danon disease prevalence and addressable patient estimates are based on available data and assumptions and are subject to uncertainty. Estimated prevalence and identified patient records do not establish the number of patients eligible for treatment. Revenue illustrations are internal modeling scenarios, rather than financial guidance, and depend on clinical success, regulatory approval, the approved patient population, patient identification, pricing, reimbursement and treatment uptake. Opportunities in additional patient populations would require further development and applicable regulatory approvals.  You should not place undue reliance on these forward-looking statements. All such statements speak only as of the date made, and Rocket undertakes no obligation to update or revise publicly any forward-  looking statements, whether as a result of new information, future events or otherwise, except as required by law. 
 

 3  © 2026 Rocket Pharmaceuticals  RP-A501 Danon Disease Program Update  Compelling development path: $1B+ commercial opportunity  Updated Phase 1 data  Evidence of disease reversal or stabilization up to 7 years post RP-A501  Initial Phase 2 data  Preliminary efficacy up to 36 months data from Cohorts 1-3  Recalibrated dose safety  Positive safety observations: 3 patients treated safely at recalibrated dose + optimized immunomodulation  Pivotal path  FDA agreement to complete Cohort 4 supporting BLA submission for accelerated approval; PRV eligible  Commercial Opportunity  10-11K US epidemiology (20K+ including EU5); 900+ Danon patients recorded in US; $1B+ global peak opportunity with initial indication 
 

 4  Catastrophic in males, with rapid progression and early mortality  Danon Males  Rapid Decline from Symptom Onset to End-Stage Heart Failure2-4  Illustrative; derived from natural history registries & cohorts  5  10  15  Age (years)  20  Cardiac Symptom Onset  Diagnosis  Loss of cardiac function  Leads to end-stage HF, life threatening  arrhythmias  Death, Heart transplant  25 30  OPTIMAL  POOR 0  Cardiac  Function  Rapid disease progression  Clinical Presentation  Hallmark Cardiac Manifestation and Potential Multi-System Involvement1  Neurocognitive  Learning  disabilities*  Mild cognitive deficits*  Visual  Retinopathy  Skeletal Muscle  Proximal muscle weakness*  Psychiatric  Anxiety  Mood Disorders  Gastrointestinal  Hepatomegaly  Elevated Transaminases  Cardiac  Left ventricular  hypertrophy*  Left ventricular dilation  Conduction abnormalities  © 2026 Rocket Pharmaceuticals  *Key symptoms that contribute to the  classical clinical triad  1. Hong KN, Eshraghian EA, Arad M, et al. J Am Coll Cardiol. 2023;82(16):1628–1647. 2. Maron BJ, Roberts WC, Arad M, et al. JAMA. 2009;301(12):1253–1259. 3. Boucek D, Jirikowic J, Taylor MRG. Genet Med. 2011;13(6):563–568.  4. Hong KN, Eshraghian E, Khedro T, et al. J Am Heart Assoc. 2025;14(7):e038394.  Danon Disease is a Highly Aggressive, Underdiagnosed, Rare Genetic Cardiomyopathy 
 

 5  PATIENT & FAMILY VOICE  I thought it would be for a couple days. But that turned into eight months.”  Mother of Danon patient - deterioration and mechanical support  while awaiting a donor heart  I’d lost both my mum and brother as they waited for transplants.”  Danon patient and emergency HTx recipient; daughter also has Danon  - 4 affected family members across 3 generations.  Transplant-Free Survival Matters: More Time Alive with Own Functioning Heart  Only ~25% ultimately receive heart transplant (HTx)  Donor scarcity & matching constraints  Diagnosis, referral & listing delays  Clinical deterioration/eligibility loss  HTx carries substantial lifelong  medical and family burden  Lifelong immunosuppression  Rejection & serious infection  Organ toxicity  Graft failure/retransplantation  HTx, heart transplantation; LVAD, left ventricular assist device;  1. Hong et al. JAHA. 2025;14:e038394. 2. ISHLT Fast Facts. 2024. 3. Hong et al. J Card Fail. 2022;28:664–669. 4. Lotan et al. Circ Genom Precis Med. 2020;13:e003117. 5. Velleca et al . J Heart Lung Transplant. 2023;42:e1–e141.  6. Royal Brompton & Harefield Hospitals, “My stor y — Caroline Earnshaw”: https://www.rbht.nhs.uk/patients-visitors/patients/patient-support-services/patient-experience-and-involvement/patient/my-story-caroline-earnshaw.  No Approved Disease-Modifying Therapy Exists  Heart transplantation is the only end-stage rescue and only for a few  HTx  HTx is lifesaving, but carries early mortality risk  HTx can be effective  10+ yrs Median survival after HTx in general  population  87.1% 5-year graft survival in Danon HTx  cohort  Early mortality remains  5 of 8 Died within 1 year after HTx/LVAD in a Danon cohort  7. Cleveland Clinic Children’s, “Heart Transplant Patient Finds New Life at Cleveland Clinic Children’s”: https://my.clevelandclinic.org/patient-stories/100-heart-transplant-patient-finds-new-life-at-cleveland-clinic-childrens.  © 2026 Rocket Pharmaceuticals 
 

 Danon Disease Biology is Exceptionally Suited to LAMP2B Gene Addition  A clear molecular defect. A direct therapeutic approach. A measurable path from biology to clinical impact.  DANON DISEASE: LAMP2B deficiency  LAMP2 Gene Addition: Restores Cellular Recycling  Diseased cardiomyocyte  cellular injury, hypertrophy and adverse remodeling  B)  X-linked  LAMP2  mutation  AAV9 with functional LAMP2B coding sequence  Gene addition to cardiomyocytes  Lysosome  Autophagosome  cellular material to be recycled  LAMP2B  restored on lysosome membrane  Autolysosome  fusion restored  Healthier Cardiomyocyte  restored cellular homeostasis & cleanup of cellular debris  Autophagosome  cellular material to be recycled  Lysosome  (little to no LAMP2  Impaired autophagosome  and lysosomal fusion  Harmful build-up accumulation of autophagic vacuoles & cellular debris  Degraded materials restored cellular recycling & clearance  Schematic; not drawn to scale. Informed by published LAMP2/LAMP2B, autophagy and AAV gene-addition biology (1-3).  1. McNally EM, Spencer MJ. N Engl J Med. 2025;392:1028–1032. 2. Argiro A, et al. JACC Heart Fail. 2024;12(2):248–260. 3. Greenberg B, et al. N Engl J Med. 2025;392:972–983.  © 2026 Rocket Pharmaceuticals  6 
 

 7  Phase 1 and Phase 2 Study of RP-A501 in Danon Disease  Global, single-arm, Pivotal Phase 2 Trial of RP-A501 ongoing; 12-Patients aligned with FDA  IM, immunomodulatory regimen; LAMP2, lysosome-associated membrane protein 2; LTFU, Long-term follow-up; LV, left ventricular; LVMI, left ventricular mass Index  1. Greenberg B., et al. N Engl J Med. 2025;392(10):972-983; 2. Gene Therapy Study of RP-A501 in Male Patients With Danon Disease. ClinicalTrials.gov identifier NCT06092034  Adult/Adolescent (n=3)  6.7 x 1013 GC/kg RP-A501  IM regimen: Steroid (n=1);  Steroid + Tacrolimus (n=2)  Adult/Adolescent (n=2)  1.1 x 1014 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Tacrolimus  Cohort 1  Cohort 2  Cohort 1A  Adult/Adolescent (n=1)  6.7 x 1013 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Sirolimus  Pediatric (n=3)+  6.7 x 1013 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Sirolimus  +Included initial sequential Pediatric Safety Run-in (n=2)  Adult/Adolescent and Pediatric with C3 inhibitor (n=2)  6.7 x 1013 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Sirolimus + C3 inhibitor  Cohort 1  Cohort 2  Cohort 3  Adult/Adolescent and Pediatric (n=12)  3.8 x 1013 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Sirolimus  Cohort 4  Pediatric (n=2)  6.7 x 1013 GC/kg RP-A501  IM regimen: Rituximab + Steroid + Sirolimus  Phase 1: 3 years (completed)  Phase 1 LTFU: up to 10 years (ongoing)  Pivotal Phase 2 (ongoing)2  Current Status:  Enrolling and treating remaining  Cohort 4 patients (n=9) in Pivotal Phase 2 Study  Co-Primary Endpoints  (at 12 months):  Improvements in LAMP2 protein expression  (≥ Grade 1 from baseline)  Reductions in Left Ventricular  Mass (LVMI; ≥10%)  Pivotal Phase 2 Trial  © 2026 Rocket Pharmaceuticals  Phase 1 Results in NEJM ‘241 
 

 8  Pre-infusion  Visit 0  Post-infusion  Visit 1  Post-infusion  Visit 2  Post-infusion  Visit 3  6  7  6.7 × 1013 GC/kg  Pediatric, N=2  M12  M24  M36  100 µm  M12  M24  M36  100 µm  2  3  6.7 × 1013 GC/kg  Adult/adolescent, N=3  M12  M24  M36  100 µm  M9  M24  M36  †Reflects 9M visit biopsy as 12M biopsy not performed.  *Grade 0 LAMP2 protein IHC staining at the 30- and 36-month assessments, however, LAMP2B vector RNA and DNA (VCN) levels have  persisted through 60 months of follow-up  ¶Grading of LAMP2 protein expression by IHC was done by a board-certified pathologist in a bl inded fashion. The semi-quantitative grading  reflects the extent of LAMP2 protein expressing cardiomyocytes in the entirety of biopsy sample according to the scale.  *Patient 5 had LV systolic dysfunction (LVEF <40%) at enrollment and had progressive heart failure requiring transplantation 5m following RP-A501 treatment and is not evaluable for efficacy.  RP-A501 Phase 1 Study: Sustained Cardiomyocyte LAMP2 Expression  Durable myocardial LAMP2 protein expression seen in all patients  Myocardial LAMP2 Protein Expression Representative LAMP2 IHC Images  BL, baseline; IHC, Immunohistochemistry; LAMP2, lysosome-associated membrane protein 2; m, month(s); Data Extraction Date: July 31, 2026  Cohort  Patient  BL  M6  M12  M18  M24  M30  M36  M60  1  0  NP  NP  0*  0*  6.7 × 1013 GC/kg  Adult/adolescent  2  0  NP  3  0  NP  †  NP  1.1 × 1014 GC/kg  Adult/adolescent*  4  0  NP  NP  6.7 × 1013 GC/kg  Pediatric  6  0  NP  NP  7  0  NP  NP  = Grade 3 (51%–74%)  = Grade 4 (≥75%)  Legend: IHC Staining Grade¶ (% Positive Cardiomyocytes)  Grade 0 = no staining = Grade 1 (≤25%)  NP = not performed = Grade 2 (26%–50%)  © 2026 Rocket Pharmaceuticals 
 

 9  Improved  Stabilized  Wor sened  © 2026 Rocket Pharmaceuticals  Cohort  Patient  Age at Most RV (y)  Most Recent  Visit (m)  Δ LVMI,*  BL to RV (g/m2.7)  Δ IVSd,  BL to RV (mm)  Δ LVPWd,  BL to RV (mm)  Δ NT-proBNP,  BL to RV (ng/L)  Δ cTnI,†  BL to RV (ng/mL)  Δ NYHA  Class  Δ KCCQ-12,  BL to RV  1:Low Dose Adult/ Adolescent  1  24  84‡  -32%,  85.0 to 57.8  +2%,  19.8 to 20.2  -30%,  18.8 to 13.2  -31%,  336 to 233  -99%,  0.60 to 0.01  II to I  +56,  44 to 100  2  26  72  -31%,  260.2 to 178.9  -29%,  60.1 to 42.6  -71%,  39.1 to 11.3  -90%,  5119 to 489  -96%,  1.46 to 0.06  II to I  +24,  64 to 88  3  23  54‡  -12%,  98.2 to 86.7  -30%,  30.9 to 21.7  -56%,  32.1 to 14.1  -45%,  841 to 460  -76%,  0.28 to 0.07  II to II  +7,  77 to 84  2:High Dose Adult/ Adolescent  4  23  36  -7%,  68.6 to 63.6*  +5%,  18.0 to 19.0  -27%,  24.0 to 17.4  -65%,  720 to 249  -39%,  0.47 to 0.29  II to I  +9,  79 to 89  3:Low Dose  Pediatric  6a  15  36  -47%,  141.5 to 74.7  -32%,  42.4 to 29.0  -2%,  22.8 to 22.3  -75%,  1629 to 406‡  -84%,  1.78 to 0.28  II to I  +13,  50 to 63  7  14  36  +3%,  82.0 to 84.9*  +13%,  18.5 to 20.9  +66%,  14.9 to 24.7  -35%,  1912 to 1238  -31%,  1.08 to 0.74  II to I  +36,  52 to 88  *Centrally evaluated (blinded) MRI data were utilized for LVMI when available. All other measurements of cardiac structure and function reflect centrally evaluated (blinded) echocardiogram data.  † Central laborator y assessment of cTnI was performed on cryopreserved and non-cryopreserved samples. Values for cTnI from high-sensitivity and earlier tests. High-sensitivity and earlier assays are expressed in ng/ m L.  ‡ For Patient 1, NYHA class and KCCQ-12 assessments occurred at Month 84, and all other assessments at Month 72. For Patient 3, NYHA class assessment occurred within the Month 84 visit window, and al l other  assessments at Month 54.  aPatient underwent heart transplant at 4.8 years post-RP-A501 infusion; post data cut.  RP-A501 Phase 1: Benefit Observed Across All Key Parameters up to 7 years  Lower cardiac biomarkers accompany stable or reduced LV mass index and improved quality-of-life scores  BL, Baseline; BNP, Brain Natriuretic Peptide; cTnI, cardiac troponin I; LAMP2, lysosome-associated membrane protein 2; IVSd, Interventricular Septum in diastole; KCCQ, Kansas City Cardiomyopathy Questionnaire; LV, Left Ventricle; LVEF, Left Ven tricular Ejection Fraction; LVMI, Left Ventricular Mass Index, LVPWd, Left Ventricular  Posterior Wall in diastole; m, month(s); MRI, magnetic reso nance imaging; NT-Pro-BN P, N-terminal pro–B-type natriuretic peptide; NYHA, New York Heart Association, RV, (Most) Recent Visit; y, year(s)  Data Extraction Date: July 31, 2026 
 

 10  Improved or Stabilized Cardiac Biomarkers and LV mass in RP-A501 Patients  Differs significantly from expanded natural history data  BNP (pg/ml)  Age [years]  N = 22 patients r2= 0.846  LV mass (g)  Age [years]  BNP increases by 42.17 ± 18.46 pg/mL per year in untreated Danon males*  LV Mass increases by 39.86 ± 4.01 g per year in untreated Danon males*  N = 20 patients r2= 0.246  Baseline  M9  M12  M18  M24  M30  M36  M42  M48  M54  M60  0  500  1000  1500  2000  Months Post Gene Therapy  BNP (ng/L)  1  2  3  4  6  7  100  6 12 18 24 30 36 42 48 54 60  Months Since RP-A501 Infusion  1000  900  800  700  600  500  400  300  200  100  0  LV mass (g)  1  2  3  4  6  7  Baseline  Months Since RP-A501 Infusion  *Unpublished data from International Danon Disease Registry  BNP, Brain Natriuretic Peptide; LV, Left Ventricle; Data Extraction Date: July 31, 2026 (Phase 1 RP-A501 study)  © 2026 Rocket Pharmaceuticals 
 

 11  -16%  -14%  -18%  -11%  -32%   -15%   RP-A501 Phase 1 Summary  LAMP2 expression and LVMI improvements seen as early as 6 months post RP-A501  * Where possible, cardiac MRI assessments shown (patients 1, 4, and 7); otherwise, echocardiogram data presented.  † Utilized 18 m data when 12m assessment was not done. ‡Reflects 9M visit biopsy as 12M biopsy not performed.  LAMP2, lysosome-associated membrane protein 2; LVMI, left ventricular mass index; MRI, magnetic resonance imaging; m, month(s). Data Extraction Date: July 31, 2026  Summary of Phase 1 in Relation to Pivotal Co-Primary Endpoints  All patients showed ≥10% LVMI decrease and LAMP2 protein expression increase (≥ Grade 1) at ~12m, representing 100% response rate based on pivotal Ph 2 endpoints  All patients showed durable myocardial LAMP2 protein expression and improved or sustained LVMI at most recent visit  M12  M18†  M18†  M12  M12  M12  LVMI % Change: Baseline to 12m†  Patient 1*  Patient 2  Patient 3  Patient 4*  Patient 6  Patient 7*  Myocardial LAMP2 Protein Expression  © 2026 Rocket Pharmaceuticals 
 

 12  RP-A501 Pivotal Phase 2 Trial Design  Pivotal, global, single-arm, open-label trial1  FDA, US Food and Drug Administration; hsTnI, high-sensitivity troponin I; LAMP2, lysosome-associated membrane protein 2; LV, left ventricular; LVMI, left ventricular mass Index; IM, immunomodulatory; NYHA, New York Heart Association;  1. Gene Therapy Study of RP-A501 in Male Patients With Danon Disease. ClinicalTrials.gov identifier NCT06092034  Pediatric and Adult/Adolescent (n=12) Recalibrated dose: 3.8 x 1013 GC/kg RP-A501  Revised IM regimen: Rituximab + Steroid + Sirolimus  Cohort 4  Key eligibility criteria Males age ≥8 years, LAMP2 mutation, NYHA II-III, evidence of LV hypertrophy, elevated hsTnI  Co-Primary Endpoint  To support accelerated approval, co-primary endpoint consisting of improvements in LAMP2 protein expression (≥ Grade 1 from baseline) and reductions in Left Ventricular Mass (LVMI; ≥10% ↓) at 12-month post-infusion  Patient 7 Patient 8 Patient 9  *Initial patients received RP-A501 at the recalibrated dose of 3.8 × 10¹³ GC/kg with a refined immunomodulatory regimen and enhanced safety monitoring. Dosing proceeded sequentially, with at least four weeks between infusions. The recalibrated Phase 2 dose was selected to deliver a therapeutic profile consistent with the dose at which RP-A501 demonstrated meaningful efficacy in Phase 1 patients.  * * *  + 9 patients (total n=12)  Cohort 1-3 completed  FDA agreement obtained to support completion of pivotal study  Pivotal data package: 12 patients at recalibrated dose + revised immunomodulatory regimen (Cohort 4)  Clinical sites ready to enroll and dose  High inbound patient interest based on favorable benefit/risk  Patients identified to support completion of clinical study  Clear path to BLA submission to support accelerated approval  © 2026 Rocket Pharmaceuticals 
 

 13  BL, Baseline; cTnI, cardiac troponin I; LAMP2, lysosome-associated membrane protein 2; IVSd, Interventricular Septum in diastole; KCCQ, Kansas City Cardiomyopathy Questionnaire; LV, Left Ventricle; LVEF, Left Ventric ular Ejection Fraction; LVMI, Left Ventricular Mass Index, LVPWd,  Left Ventricular Posterior Wall in diastole; m, month(s); NT-Pro-BNP, N-terminal pro–B-type natriuretic peptide; NYHA, New Yor k Heart Association, RV, (Most) Recent Visit; y, year(s)  Data Extraction Date: August 7, 2026  Improved  Stabilized  Worsened  Cohort  Patient  Age at Most RV (y)  Most Recent Visit (m)  Δ LVMI,  BL to RV (g/m2.7)  Δ IVSd,  BL to RV (mm)  Δ LVPWd,  BL to RV (mm)  Δ NT-proBNP,  BL to RV  (ng/L)  Δ cTnI,  BL to RV (ng/mL)  Δ NYHA  Class  Δ KCCQ-12 OS,  BL to RV  Cohort 1: Adult/ Adolescent  2-1  20  24  -36%,  65.5 to 41.9  -28%,  21.8 to 15.6  -7%,  16.5 to 15.4  -19%,  125 to 101  -49%,  0.04 to 0.02  II to I  0,  100 to 100  Cohort 2:  Pediatric  2-2  16  36  -35%,  65.4 to 42.8  -39%,  16.0 to 9.7  -10%,  10.4 to 9.4  -7%,  54 to 50  -14%,  0.02 to 0.01  II to I  +2,  98 to 100  2-3  15  24  +9%,  187.1 to 204.8  -4%,  28.2 to 27.1  -17%,  26.1 to 21.7  -19%,  7966 to 6465  +122%,  0.33 to 0.73  III to I  +16,  64 to 79  2-4  12  18  -30%,  202.2 to 140.5  +17%,  39.3 to 46.0  +24%,  22.5 to 27.9  -30%,  69855 to 49135  -14%,  0.82 to 0.71  II to II  +35,  35 to 71  Preliminary Efficacy of Phase 2 Study RP-A501: Up to 36 months  Preliminary findings show stabilization or improvement in key disease measures  All measurements of cardiac structure and function (LVEF, LV Mass, LVMI, IVSd and LVPWd) utilized centrally evaluated echocardiogram data (blinded review) for all participants.  In Phase 2 Cohort 3, two patients treated with immunomodulatory regimen containing a C3-inhibitor experienced serious adverse events.  Cohort 3 is discontinued and data from the two subjects were not included.  Cohort 4 Updates and Rationale  Dose recalibrated to account for the higher full-to-empty capsid ratio  Immunomodulation revised based on early Phase 2 safety findings  © 2026 Rocket Pharmaceuticals 
 

 Modified Protocol Delivers Favorable Pivotal Cohort 4 Safety Profile  aPatients 2-3, 2-4 and 2-6; bPatient 2-8.  GGT, Gamma-glutamyltransferase; IM, immunomodulatory; TEAE, Treatment Emergent Adverse Events  Phase 2  Preferred Term (Grade ≥3)  Cohorts 1 & 2 (n=4)  Cohort 3 (n=2)  Cohort 4 (n=3)  Subject with at least 1 Serious TEAE (Grade  ≥3)  3 (75.0)  2 (100)  1 (33.3)  Thrombotic microangiopathy a  2 (50.0)  1 (50.0)  0  Acute kidney injury  1 (25.0)  1 (50.0)  0  Renal failure  1 (25.0)  0  0  Gamma-glutamyltransferase increased b  0  0  1 (33.3)  Hepatic enzyme increased  1 (25.0)  0  0  Cardiac arrest  1 (25.0)  0  0  Posterior reversible encephalopathy  syndrome  1 (25.0)  0  0  Rhabdomyolysis  1 (25.0)  0  0  Aplastic anemia  0  1 (50.0)  0  Disseminated intravascular coagulation  0  1 (50.0)  0  Cytokine release syndrome  0  1 (50.0)  0  Hypertransaminasaemia  0  1 (50.0)  0  Pneumothorax  0  1 (50.0)  0  Sepsis  0  1 (50.0)  0  Shock  0  1 (50.0)  0  Retroperitoneal hemorrhage  0  1 (50.0)  0  Grade ≥3 Serious Treatment Emergent Adverse Events Related to RP-A501  Pivotal Cohort 4  (recalibrated dose with modified IM regimen)  14  Data Extraction Date: Aug 7, 2026  © 2026 Rocket Pharmaceuticals  No evidence of thrombotic microangiopathy, capillary leak or other complement-mediated events  Elevated GGT observed in 1 patient; resolved and patient discharged after 12 days.  All 3 patients discharged by 1-2 weeks post-infusion 
 

 No Evidence of TMA,CLS, or Other Severe Complement-Mediated Toxicities in Cohort 4  Platelets and Terminal Complement Complex (sC5b9) of the initial 3 patients up to 3 months post-treatment  CLS, Capillary Leak Syndrome; sC5b9, soluble C5b9; TMA, Thrombotic microangiopathy  ‡  ‡ A decrease in platelet count was observed at Week 8-9 in the absence of symptoms or signs suggestive of platelet dysfunction or deficit. All concomitant medications were reviewed to discontinue any  potential contributors; it was not deemed clinically significant by the investigator and Sponsor and resolved following medication modifications.  *The sC5b9 lower limit of detection per the validated assay at the clinical site is <170 ng/mL; reported values of <170 ng/mL are shown as 170 ng/mL.  Patient 2-7  Patient 2-8  Patient 2-9  Normal Range  Normal Range  15  Data Extraction Date: September 4, 2026  © 2026 Rocket Pharmaceuticals  Platelets  Complement Marker (sC5b9) 
 

 16  PHASE 1 DURABILITY  Up to 7 Years  of clinical benefit observed  Clinical benefit and improvements observed with durable biomarker in all patients at latest trial follow-up  100% overall survival; median transplant-free survival not reached at median follow-up: 5.7 years (range: 4.3–7.1 years)  Mirrors Phase 1 Trajectory  Improvement or stabilization of cardiac hypertrophy, heart failure symptoms, quality of life scores, and biomarkers of cardiac injury and stress replicate the trajectory of Phase 1 responders  Generally Well-Tolerated  Recent protocol optimizations suggest successful mitigation of complement-mediated toxicities*  *In Phase 2 Cohort 3, two patients treated with immunomodulatory regimen containing a C3-inhibitor experienced serious adverse events. The cohort is discontinued and data from the two subjects were not included.  RP-A501 Demonstrated Durable Efficacy with a Tolerable Safety Profile  SAFETY AND TOLERABILITY  PHASE 2 EARLY EFFICACY  © 2026 Rocket Pharmaceuticals 
 

 17  RP-A501 Pivotal Phase 2 Milestones  TODAY  Enrollment  and dosing  9 additional patients  in Cohort 4  MID-2027  Dosing  completion  Last patient dosed 12-month follow-up  MID-2028  TOPLINE TARGET*  Primary efficacy readout from pivotal Cohort 4  LATE-2028  BLA regulatory  filing  PRV eligible; Accelerated Approval; Priority review  CO-PRIMARY EFFICACY ENDPOINTS AT 12 MONTHS  © 2026 Rocket Pharmaceuticals  ≥ Grade 1  Improvement in LAMP2  protein expression from baseline  ≥10%  Reduction in left ventricular  mass index (LVMI)  *MID-2028 target is provisional pending alignment with dosing completion and 12-month follow-up. 
 

 18  Schematic Male Disease Progression  HTX Danon Patients  HCM Symptomatic  Danon Patients  Pre-HCM  Danon  Patients  Estimated Epidemiology of Danon Disease is 20K+ across US & EU5  Independent genotype and phenotype-based analyses converge on similar estimate of 10-11K US patients  Genotype-Based Analysis  Pathogenic & likely Pathogenic LAMP2 Variants  Based on comprehensive molecular characterization of confirmed  Danon Disease + population genomic anchor  Classic loss-of-function LAMP2  variants  Population allele frequency of 3 variant  categories1 from gnomAD2  Other P/LP LAMP2 variants  6 variant categories3 identified through RW  datasets4 with confirmed DD  Phenotype-Based Analysis  Natural History and Real-World Modeling  Based on Danon disease natural history modeling + real-world  patient data  Patients with HCM Phenotype5  Age & sex-specific DD yield modeling with HCM population  Pre-cardiomyopathy and DCM  Patients  ~5.4K  Age- and sex-specific  natural history model built  with 700+ patient data  DCM, dilated cardiomyopathy; DD, Danon disease; EU5; Germany, France, Ital y, Spain, UK; HCM, hypertrophic cardiomyopathy; HTx, heart transplantation; P/LP, pathogenic/likely pathogenic; RW, real-world;  1. S top-gain (Nonsense), Frameshift and Canonical splice-site variant categories 2. non-UKBB gnom AD v4.11 1. non-UKBB gnom AD v4.11 Genome Aggregation Database 3. includes Start-loss, Exon/gene deletions/duplications, Missense, Synonymous and Intronic variant categories 4. variant records of confirmed DD patients from genetic testing labs Invi tae, Ambry Genetics and GeneDx, real-world Natural History Cohor ts, Rocket Natural History S tudy, Reposi tory of published RW Danon patient cases 5. HCM & DD claims/EHR databases (Cellworks, Forian) covering >310M lives  ESTIMATED DANON POPULATION  10-11K  PATIENTS  © 2026 Rocket Pharmaceuticals  ~6-7K  Female  ~4K  Male  62%  (~6.6K)  Anchor  38%  (~4.1K)  Scaling  ~5.1K  Anchor  Scaling  450+ patient records  9+ variant categories 
 

 19  Over 900 Identified US Danon Disease Patients  Representing a current diagnosis rate under 10%  CM, Cardiomyopathies; DD, Danon disease; HCM, hypertrophic cardiomyopathy; HTx, heart transplantation  1. Data as of July 2026; over 80% of patients reported in ICD-10 code and genetic testing lab data have been identified in the last 5 years; ICD-10 code for Danon available from Q 4 2023. 2. Non-sponsored de-identified test data purchased thr ough commercial genetic testing labs; 3. Does not include VUS patients; business rules for de-identification and de-duplication; 4. Estimated based on Danon true prevalence modeling and patients found; 5. Longoni M, et al Real-world util ization of guideline-directed genetic testing in inherited cardiovascular diseases; 6. Bui QM, et al. Real-world Genetic Testing Practices in Cardiomyopathy, 2026  Gender  45%  55%  Institutional coverage (% of pts)  30%  60%  100%  Top 25  Top 100  All Institutions  (n=290)  Est. DD Diagnosis Rate4  <10%  Genetic Testing Rates in Cardiomyopathies (%)  1.6%  2.6%  13.8%  Adult HCM5  Adults with  CM (<=40)6  Pediatric CM6  Sources1  Claims/  EHR  Field  Effort  900  Genetic HCP  Test Data2 Reported  De-duplicated for unique3 patient counts  ≥15 patients 5-14 patients 2-4 patients 1 patient  Patients cumulatively identified and include those who received HTx or subsequently experienced disease-related mortality  © 2026 Rocket Pharmaceuticals 
 

 20  Accelerating Danon Diagnosis: Focused Path to Launch  Aspiration: every person with HCM or a Danon disease signature and every at-risk relative of a confirmed Danon patient is genetically evaluated so no Danon diagnosis is missed  Illustrative Diagnoses Ramp  External tailwinds   HCM recognition & CMI entry into pediatrics   Ecosystem momentum around cardio genomics & broader testing   Genotype-driven trials in cardiomyopathies  <10%  At launch (Mid-2029)  Longer term  Diagnosed Share of Target Population  Drive cardiologists to test young HCM & urgency with Danon signature  Expand diagnostic infrastructure  & access  Empower patients & caregivers to seek genetic evaluation  Multiply diagnoses through the family cascade  HCM, hypertrophic cardiomyopathy; CMI, Cardiac myosin inhibitor s  © 2026 Rocket Pharmaceuticals 
 

 21  Addressable Opportunity in Danon Extends Beyond Males with HCM  Expand the window, extend the impact, transform the disease  HCM, hypertrophic cardiomyopathy; Illustrative, non-risk-adjusted peak annual commercial opportunity based on internal assumptions regarding addressable populations, diagnosis, treatment eligibility, pricing, access and uptake. Potential lifecycle expansions require additional clinical evidence and regulatory alignment and approval. Actual indications, eligiblepopulations, timing and commercial outcomes may differ. Segment sizes are not drawn to scale.  Danon Females with earlier-onset HCM  Danon Males with HCM  $1B+  GLOBAL PEAK  ANNUAL OPPORTUNITY  UNLOCK DANON OPPORTUNITY  ◷  Treat earlier  Shift treatment upstream  Capture fast progressors Intervene before irreversible progression  ◎  Reach underdiagnosed females  Treat patients historically missed  Danon Males pre-HCM  Danon Females with later-onset Cardiomyopathy  Danon gene therapy addressable segment  $2B+  GLOBAL PEAK ANNUAL  OPPORTUNITY  © 2026 Rocket Pharmaceuticals 
 

 22  New Evidence and a Defined Pivotal Path  Sustained Clinical Benefit  RP-A501 conferred durable disease reversal/stabilization through  36 months and up to 7 years post-RP-A501 infusion  Preliminary efficacy in initial 4 patients in Phase 2 further  corroborates long-term benefit identified in Phase 1 and LTFU  Early safety at the recalibrated dose  No TMA, capillary leak, or significant complement-mediated adverse events through 3 months in the first three patients  Attractive Commercial Opportunity  US epidemiology of 10-11K US (20K including Europe); over 900 Danon disease patients recorded in US alone; $1B global peak potential for 1st indication  Efficacy target  At least  7 of 12  responders at 12 months  Each responder must meet both criteria  Myocardial LAMP2 expression ≥ Grade 1 from baseline LVMI reduction ≥10% from baseline  © 2026 Rocket Pharmaceuticals  Dosing completion expected mid-2027  Current cash runway now into Q3’28  RP-A501 is investigational. Initial safety follow-up is limited for Phase 2 Cohort 4. One RP-A501-related Grade 3 increase in gamma-glutamyltransferase (GGT) was reported and resolved. Safety data  as of September 4, 2026. Approval requires FDA review of the complete application. 
 

 


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